Research And Grants

Multilamellar H3K27M RNA Aggregates Targeting Diffuse Midline Glioma

Diffuse midline gliomas (DMGs) are some of the worst childhood cancers, typically marked by an H3K27M mutation, which is an excellent target as it is expressed by nearly all the tumor cells. 

We have designed a way to repetitively layer H3K27M attack ‘orders’ in an “onion-like” nanoparticle, which intensely and rapidly stimulates the immune system. This approach cured a large percentage of mice harboring DMGs and, in dogs and adults, has generated remarkable immune responses. In addition, as an off-the-shelf treatment, this vaccine is rapidly available and much easier to give than treatments which require prolonged times for generation. Rapidly retraining the immune system against H3K27M has great potential to improve outcomes for a desperate group of children and their families.

We are hoping to try this in a clinical trial for children with H3K27M+ DMG after standard local radiation treatment. This funding request encompasses the first arm who will receive a year of vaccine therapy. In addition, blood will be drawn so that scientists can examine how the patient’s immune system is responding to the H3K27M nanoparticle vaccine. With a successful trial, we anticipate rapid translation into a Phase 2 combination trial.